Orkambi

"This transaction supercharges our efforts to develop lifesaving therapies in order to pursue daring, new opportunities that one day may lead to a permanent, lifelong cure for this disease.”

Robert Beall, PhD, President and CEO, Cystic Fibrosis Foundation.

Approved applications

ORKAMBI™ is an FDA-approved combination therapy for the treatment of cystic fibrosis (CF).

ORKAMBI™ is a combination of two categories of molecules, FDA-approved ivacaftor (known by its brand-name KALYDECO) and experimental therapy lumacaftor (known also as VX-809), both of which help in correcting the mutated genes in patients with cystic fibrosis – a novel therapeutic approach designed to treat the root cause of cystic fibrosis instead of treating the severity of symptoms. Both ivacaftor and lumacaftor work by correcting the misfolded CFTR protein, which is the root cause of the F508del mutation. ORKAMBI™ has been approved for people ages 12 and older who have two copies of the F508del mutation -- the most common cystic fibrosis mutation. The F508del mutation causes a series of problems that prevent CFTR from achieving the correct shape and reaching the cell surface. The combination drug treats these problems with a two-step approach: Lumacaftor helps move the defective CFTR protein to its proper place at the cell surface. Ivacaftor increases the activity of the protein once it is there, supporting the flow of salt and fluids, which helps thin the thick mucus that builds up in the lungs and other organs.